Higher efficiency of thymine-adenine clamp-modified single-stranded oligonucleotides in targeted nucleotide sequence correction is not correlated with lower intracellular degradation

Human Gene Therapy

Volumn 20, Issue 3, 2009, Pages 283-287

  Wuepping, M ^a   Kenner, O ^a   Hegele, H ^a   Schwandt, S ^a   Kaufmann, D ^a  

^a UNIVERSITY OF ULM   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

ADENINE; CYTOSINE; GUANINE; HYPOXANTHINE PHOSPHORIBOSYLTRANSFERASE; OLIGONUCLEOTIDE; PHOSPHOROTHIOIC ACID; THYMINE;

ANIMAL CELL; ARTICLE; CELL METABOLISM; CONTROLLED STUDY; CORRELATION ANALYSIS; CYTOPLASM; EUKARYOTE; NONHUMAN; NUCLEOTIDE SEQUENCE; POINT MUTATION; PROTEIN CLEAVAGE; PROTEIN DEGRADATION; PROTEIN STABILITY; PROTEIN TARGETING; SEQUENCE ANALYSIS;

ADENINE; ANIMALS; BASE SEQUENCE; CELL LINE; CRICETINAE; DNA, SINGLE-STRANDED; HYPOXANTHINE PHOSPHORIBOSYLTRANSFERASE; INTRACELLULAR SPACE; MOLECULAR SEQUENCE DATA; PHOSPHOROTHIOATE OLIGONUCLEOTIDES; POINT MUTATION; REPRODUCIBILITY OF RESULTS; TARGETED GENE REPAIR; THYMINE; TRANSFECTION;

EUKARYOTA;

EID: 62549129495     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2008.138     Document Type: Article

References (20)

1. Andrieu-Soler, C., Casas, M., Faussat, A.M., Gandpolphe, C., Doat, M., Tempe, D., Giovangeli, C., Behar-Cohen, F., and Concordet, J.P. (2005). Stable transmission of targeted gene modification using single-stranded oligonucleotides with flanking LNAs. Nucleic Acids Res. 12, 3733-3742.
2. Gamper, H.B., Parekh, H., Rice, M.C., Bruner, M., Youkey, H., and Kmiec, E.B. (2000). The DNA strand of chimeric RNA/DNA oligonucleotides can direct gene repair/conversion activity in mammalian and plant cell-free extracts. Nucleic Acids Res. 21, 4332-4339.
3. Goukassian, D.A., Helms, E., van Steeg, H., van Oostrom, C., Bhawan, J., and Gilchrest, B.A. (2004). Topical DNA oligonucleotide therapy reduces UV-induced mutations and photocarcinogenesis in hairless mice. Proc. Natl. Acad. Sci. U.S.A. 101, 3933-3938.
4. Hegele, H., Wuepping, M., Ref, C., Kenner, O., and Kaufmann, D. (2008). Simultaneous targeted exchange of two nucleotides by single-stranded oligonucleotides clusters within a region of about fourteen nucleotides. BMC Mol. Biol. 9, 14.
5. Hu, Y., Parekh-Olmedo, H., Drury, M., Skogen, M., and Kmiec, E.B. (2005). Reaction parameters of targeted gene repair in mammalian cells. Mol. Biotechnol. 3, 197-210.
6. Igoucheva, O., Alexeev, V., and Yoon, K. (2001). Targeted gene correction by small single-stranded oligonucleotides in mammalian cells. Gene Ther. 5, 391-399.
7. Igoucheva, O., Alexeev, V., and Yoon, K. (2004). Mechanism of gene repair open for discussion. Oligonucleotides 4, 311-321.
8. Igoucheva, O., Alexeev, V., Scharer, O., and Yoon, K. (2006a). Involvement of ERCC1/XPF and XPG in oligodeoxynucleotide-directed gene modification. Oligonucleotides 1, 94-104.
9. Igoucheva, O., Alexeev, V., and Yoon, K. (2006b). Differential cellular responses to exogenous DNA in mammalian cells and its effect on oligonucleotide-directed gene modification. Gene Ther. 3, 266-275.
10. Kenner, O., Kneisel, A., Klingler, J., Bartelt, B., Speit, G., Vogel, W., and Kaufmann, D. (2002). Targeted gene correction of hprt mutations by 45 base single-stranded oligonucleotides. Biochem. Biophys. Res. Commun. 5, 787-792.
11. Kenner, O., Lutomska, A., Speit, G., Vogel, W., and Kaufmann, D. (2004). Concurrent targeted exchange of three bases in mammalian hprt by oligonucleotides. Biochem. Biophys. Res. Commun. 4, 1017-1023.
12. Kmiec, E.B. (2003). Targeted gene repair: In the arena. J. Clin. Invest. 5, 632-636.
13. Liu, L., Parekh-Olmedo, H., and Kmiec, E.B. (2003). The development and regulation of gene repair. Nat. Rev. Genet. 9, 679-689.
14. Olsen, P.A., Randol, M., and Krauss, S. (2005a). Implications of cell cycle progression on functional sequence correction by short single-stranded DNA oligonucleotides. Gene Ther. 6, 546-551.
15. Olsen, P.A., Randol, M., Luna, L., Brown, T., and Krauss, S. (2005b). Genomic sequence correction by single-stranded DNA oligonucleotides: Role of DNA synthesis and chemical modifications of the oligonucleotide ends. J. Gene Med. 12, 1534-1544.
16. Parekh-Olmedo, H., Drury, M., and Kmiec, E.B. (2002). Targeted nucleotide exchange in Saccharomyces cerevisiae directed by short oligonucleotides containing locked nucleic acids. Chem. Biol. 10, 1073-1084.
17. Parekh-Olmedo, H., Ferrara, L., Brachman, E., and Kmiec, E.B. (2005). Gene therapy progress and prospects: Targeted gene repair. Gene Ther. 8, 639-646.
18. Radecke, F., Peter, I., Radecke, S., Gellhaus, K., Schwarz, K., and Cathomen, T. (2006a). Targeted chromosomal gene modification in human cells by single-stranded oligodeoxynucleotides in the presence of a DNA double-strand break. Mol. Ther. 14, 798-808.
19. Radecke, S., Radecke, F., Peter, I., and Schwarz, K. (2006b). Physical incorporation of a single-stranded oligodeoxynucleotide during targeted repair of a human chromosomal locus. J. Gene Med. 2, 217-228.
20. Sorensen, C.B., Krogsdam, A.M., Aandersen, M.S., Kristiansen, K., Bolund, L., and Jensen, T.G. (2005). Site-specific strand bias in gene correction using single-stranded oligonucleotides. J. Mol. Med. 1, 39-49.